Nadofaragene firadenovec
| Gene therapy | |
|---|---|
| Target gene | Interferon alfa-2b |
| Clinical data | |
| Trade names | Adstiladrin |
| Other names | Nadofaragene firadenovec-vncg, Instilidrin, rAd-IFN/Syn3 |
| License data | |
| Routes of administration | Intravesical |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| UNII | |
| KEGG | |
Nadofaragene firadenovec, sold under the brand name Adstiladrin, is a gene therapy for the treatment of bladder cancer.[1][3][4] It is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy.[3]
The most common adverse events including laboratory abnormalities, include increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition urgency, increased creatinine, hematuria, decreased phosphate, chills, dysuria, and pyrexia.[4]
Nadofaragene firadenovec was approved for medical use in the United States in December 2022.[3][4][5]
Medical uses
[edit]Nadofaragene firadenovec is indicated for the treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.[1][3]
History
[edit]The safety and effectiveness of nadofaragene firadenovec was evaluated in a multicenter clinical study (Study CS-003 (NCT02773849)) that included 157 participants with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer, 98 of whom had BCG-unresponsive carcinoma in situ with or without papillary tumors and could be evaluated for response.[3][4] Participants received nadofaragene firadenovec once every three months for up to twelve months, or until unacceptable toxicity to therapy or recurrent high-grade non-muscle-invasive bladder cancer.[3][4] Overall, 51% of enrolled participants using nadofaragene firadenovec therapy achieved a complete response (the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine).[3] The median duration of response was 9.7 months.[3] Forty-six percent of responding participants remained in complete response for at least one year.[3] The major efficacy outcome measures were complete response at any time and duration of response.[4] Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology.[4] Random bladder biopsies of five sites were conducted in participants remaining in complete response at twelve months.[4]
The US Food and Drug Administration (FDA) granted the application for nadofaragene firadenovec priority review, breakthrough therapy, fast track, and orphan drug designations.[3][4] The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S.[3]
Society and culture
[edit]Names
[edit]Nadofaragene firadenovec is the international nonproprietary name (INN).[6]
References
[edit]- ^ a b c "Archived copy". Food and Drug Administration. Archived from the original on 17 December 2022. Retrieved 17 December 2022.
{{cite web}}: CS1 maint: archived copy as title (link) - ^ "Adstiladrin". U.S. Food and Drug Administration (FDA). 16 December 2022. STN: BLA 125700. Archived from the original on 17 December 2022. Retrieved 16 December 2022.
- ^ a b c d e f g h i j k This article incorporates text from this source, which is in the public domain.
- ^ a b c d e f g h i This article incorporates text from this source, which is in the public domain.
- ^ "Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer". Ferring Pharmaceuticals USA (Press release). 16 December 2022. Archived from the original on 17 December 2022. Retrieved 16 December 2022.
- ^ World Health Organization (2018). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 79". WHO Drug Information. 32 (1). hdl:10665/330941.
External links
[edit]- Clinical trial number NCT02773849 for "Adstiladrin (Instilidrin) in Patients With High Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)" at ClinicalTrials.gov
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